Why the study?
Following accelerated FDA approval of eteplirsen based on dystrophin production, this study aimed to evaluate the efficacy and safety of eteplirsen in a larger cohort.
Does eteplirsen improve dystrophin production and attenuate disease progression in ambulatory patients aged 7-16 years with Duchenne muscular dystrophy amenable to exon 51 skipping?
Population
Ambulatory patients aged 7–16 years with Duchenne muscular dystrophy (79 eteplirsen-treated and 30 untreated)
Comparison
Intravenous eteplirsen 30 mg/kg/week vs untreated cohort
Design
Phase 3 multicenter open-label study
Follow-up
96 weeks
Authors
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May slow FVC decline in exon 51 DMD; leaves open need for randomized confirmation before practice change.
Does eteplirsen improve dystrophin production and attenuate disease progression in ambulatory patients aged 7-16 years with Duchenne muscular dystrophy amenable to exon 51 skipping?
Eteplirsen increases dystrophin production and may slow pulmonary and motor decline in patients with DMD amenable to exon 51 skipping compared to natural history.
McDonald et al. (2021) studied this question.
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